A phase 2 trial of neural stem cell transplantation was mostly well tolerated, with about half the patients responding to the treatment

Article Highlights:

Neuralstem is testing its NSI-566 cells, derived from human spinal cord tissue, in ALS patients; the cells are transplanted directly into the spinal cord in a surgical procedure.

In a phase 2 trial, the cells appeared well tolerated, although one trial participant experienced a serious adverse event associated with the transplantation procedure.

Seven out of the 15 participants in the phase 2 trial showed near-zero decline or improvement in either grip strength or a measure of overall function (the ALSFRS score).

Maryland-based biotechnology company Neuralstem announced in a March 12, 2014, press release, that a phase 2 trial of its NSI-566 neural stem cells in 15 people with amyotrophic lateral sclerosis (ALS) has shown that the surgical transplantation procedure and the cells themselves, up to a dose of 16 million cells, appeared to be safe and well tolerated. One trial participant expereinced a serious adverse event associated with the transplantation surgery.

Neuralstem's NSI-566 cells are derived from human spinal cord tissue. The hope is that these cells will integrate into the spinal cord, where they will protect motor neurons (muscle-controlling nerve cells) and ultimately stabilize or improve motor function.

The company said that, nine months after a surgical procedure to transplant the cells into patients' spinal cords, seven out of 15 (47 percent) of patients showed a positive response to the procedure in terms of their grip strength or their overall function on the ALS Functional Rating Scale (ALSFRS).

A positive response on grip strength testing or ALSFRS testing was defined as either a near-zero decline or an improvement.

"The top-line [preliminary] data look very positive and encouraging," said Jonathan Glass, director of the MDA ALS Center at Emory University, an investigator at one of three centers where the trial was conducted. "If this proportion of patients doing well after treatment can be corroborated in future therapeutic trials, it will be better than any response seen in any previous ALS trials. Elucidating which factors define a patient who may have a therapeutic response to the stem cell treatment will be the next key challenge. We are hopeful that a set of predictive algorithms can be established to help pre-select the responders in our future trial."